JP2023545433A - ファブリー病の治療のための組成物及び方法 - Google Patents

ファブリー病の治療のための組成物及び方法 Download PDF

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JP2023545433A
JP2023545433A JP2023521706A JP2023521706A JP2023545433A JP 2023545433 A JP2023545433 A JP 2023545433A JP 2023521706 A JP2023521706 A JP 2023521706A JP 2023521706 A JP2023521706 A JP 2023521706A JP 2023545433 A JP2023545433 A JP 2023545433A
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hgla
gla
seq
sequence
mice
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JP2023545433A5 (enExample
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オルドー，ジュリエット
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ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
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    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022—Alpha-galactosidase (3.2.1.22)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2023521706A 2020-10-09 2021-10-08 ファブリー病の治療のための組成物及び方法 Pending JP2023545433A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202063089850P 2020-10-09 2020-10-09
US63/089,850 2020-10-09
US202163146286P 2021-02-05 2021-02-05
US63/146,286 2021-02-05
US202163186092P 2021-05-08 2021-05-08
US63/186,092 2021-05-08
PCT/US2021/054145 WO2022076803A1 (en) 2020-10-09 2021-10-08 Compositions and methods for treatment of fabry disease

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JP2023545433A true JP2023545433A (ja) 2023-10-30
JP2023545433A5 JP2023545433A5 (enExample) 2024-10-10

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US (1) US20230365955A1 (enExample)
EP (1) EP4225906A1 (enExample)
JP (1) JP2023545433A (enExample)
KR (1) KR20230118075A (enExample)
AU (1) AU2021356684A1 (enExample)
CA (1) CA3193833A1 (enExample)
TW (1) TW202229560A (enExample)
WO (1) WO2022076803A1 (enExample)

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AR124981A1 (es) * 2021-02-26 2023-05-24 Takeda Pharmaceuticals Co Composición y métodos para el tratamiento de la enfermedad de fabry
US20240408157A1 (en) * 2021-12-02 2024-12-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
CA3261151A1 (en) * 2022-07-08 2024-01-11 Cornell University PHARMACEUTICAL METHODS AND COMPOSITIONS FOR THE TREATMENT AND PREVENTION OF ALZHEIMER'S DISEASE
KR20250052387A (ko) * 2022-08-25 2025-04-18 다케다 야쿠힌 고교 가부시키가이샤 파브리병의 치료에서의 사용을 위한 조성물
EP4634394A2 (en) 2022-12-17 2025-10-22 The Trustees of The University of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
WO2025007046A1 (en) 2023-06-29 2025-01-02 The Trustees Of The University Of Pennsylvania Mutant aav with central nervous system targeting motifs and compositions containing same
KR20260114566A (ko) 2023-11-14 2026-07-22 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 심근 및 골격근-특이적 표적화 모티프를 갖는 조성물 및 이의 용도

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WO2020132455A1 (en) * 2018-12-21 2020-06-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2020142752A1 (en) * 2019-01-04 2020-07-09 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of fabry disease

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WO2020077114A2 (en) * 2018-10-10 2020-04-16 Amicus Therapeutics, Inc. Disulfide bond stabilized polypeptide compositions and methods of use
WO2020132455A1 (en) * 2018-12-21 2020-06-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2020142752A1 (en) * 2019-01-04 2020-07-09 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of fabry disease

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KR20230118075A (ko) 2023-08-10
AU2021356684A1 (en) 2023-05-11
WO2022076803A1 (en) 2022-04-14
EP4225906A1 (en) 2023-08-16
CA3193833A1 (en) 2022-04-14
TW202229560A (zh) 2022-08-01
US20230365955A1 (en) 2023-11-16
WO2022076803A8 (en) 2022-06-09

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